Long-term outcomes after autologous stem cell transplantation for multiple myeloma

Abstract

As multiple myeloma (MM) treatments evolve, frequent updates are required to monitor the long-term effect of changes in approach. Traditionally, MM is considered an incurable disease, with most patients eventually relapsing. However, improvements in treatments has raised the possibility that MM might be functionally curable. To examine improvements in long-term survival, we followed 4329 patients with newly diagnosed MM treated with autologous stem cell transplantation (ASCT) at the University of Arkansas for Medical Sciences from 1989 through 2018. Overall survival (OS) and progression-free survival (PFS) were evaluated using Kaplan-Meier analysis, Cox proportional hazards models, relative survival analysis, and cure modeling among different time periods, risk groups, and demographic traits. Steady improvements in OS were found, with patients treated in 2014 or later having superior OS (hazard ratio, 0.35; 95% confidence interval [CI], 0.27-0.45) and reduced excess risk for MM death (relative excess risk, 0.30; 95% CI, 0.22-0.41) compared with patients treated in 1997 or earlier. Patients treated during intervening time periods often had intermediate survival, but trends in OS, PFS, and landmarked analyses were inconsistent. Cure models support the potential for cure, ranging from 6.3% to 31.3%, depending on the year of treatment, with 10.0% to 18.6% of patients achieving their normal life expectancy across multiple periods. There was some evidence of reductions in early mortality within 3 years of diagnosis, longer complete response (CR) duration, and reductions in relapse after achieving CR. However, results differed depending on age, risk group, and cytogenetic characteristics.

Role of Stem Cell Transplantation in Multiple Myeloma

“…Given the exorbitant prices of the novel drugs, early ASCT could reduce the financial burden to the patient. ASCT has unquestionable efficacy, and it confers a complete remission in one-third of transplant recipients and a median PFS of 18–27 months even without any further therapy [ 64 ]. It is a relatively safe procedure with a transplant-related mortality of <1% in experienced centers [ 65 ].…”

Prognostic and Predictive Factors in Newly Diagnosed Multiple Myeloma Patients with Early Mortality with Prediction Matrix and Three and Five-Year Overall Survival

“…Currently, the use of venetoclax was stopped due to an early signal for increased death in early clinical trials due to a higher rate of infections [54]. A large, US, multicenter prospective observational cohort study did not demonstrate any impact of t (11;14) on PFS, or OS [55]. Further clinical trials investigating its use in myeloma are currently pending.…”

“…Second stem cell transplantation may be considered in those with progression-free survival (PFS) or more than three years. Similarly models have supported the potential for cure, estimated at 6.3% to 31.3% depending on the year of treatment [55]. Whether novel agents will supplant HDT followed by SCT backed by minimal residual disease (MRD) continues to be explored.…”

The effect of pamidronate delivery in bisphosphonate-naïve patients on neutrophil chemotaxis and oxidative burst

“…Bone is an organ that is frequently affected by the metastatic spread of several cancers 1 . Multiple myeloma, an incurable malignant plasma cell dyscrasia, is associated with bone metastases in approximately 90% of those patients diagnosed with this condition 2 – 4 . The pathogenesis of multiple myeloma is characterized by increased osteoclastic activity and osteoblast dysfunction which lead to localized bone resorption and skeletal-related events (SREs) 2 .…”